Related Experiment Video
Updated: Dec 30, 2025
12:57
Investigating the Spreading and Toxicity of Prion-like Proteins Using the Metazoan Model Organism C. elegans
Published on: January 8, 2015
16.4K
Cellular models for discovering prion disease therapeutics: Progress and challenges
Saffire H Krance1,2, Russell Luke3, Marc Shenouda4,5
1Department of Pharmacology and Toxicology, University of Toronto, Toronto, ON, Canada.
Journal of Neurochemistry
|January 17, 2020
Summary
Discovering effective prion disease treatments is challenging. Cellular models are crucial for identifying anti-prion drugs, but limited models for human prions hinder Creutzfeldt-Jakob disease therapy development.
Area of Science:
- Neurodegenerative diseases
- Protein misfolding disorders
- Infectious protein aggregates
Background:
- Prion diseases, like Creutzfeldt-Jakob disease, are fatal neurodegenerative disorders caused by misfolded, infectious proteins.
- Currently, no treatments exist to halt or slow prion disease progression in the brain.
- Animal models are vital for prion disease research, but cell culture models offer a more powerful system for drug discovery.
Purpose of the Study:
- To review available cellular models for discovering and testing anti-prion drugs.
- To highlight challenges in developing therapies for prion diseases, particularly those caused by human prions.
Main Methods:
- Utilizing chronically infected cell lines to propagate various prion types.
- Testing candidate anti-prion drugs for efficacy in cellular models.
- Evaluating the effectiveness of drugs against different prion species.
Main Results:
- Cellular models have identified potential anti-prion drugs partially effective in rodents.
- Drug efficacy varies significantly between different prion strains and species.
- Developing cell culture models for non-mouse, especially human, prions remains a significant hurdle.
Conclusions:
- Cellular models are essential for identifying prion propagation inhibitors.
- Limited models for human prions impede the discovery of Creutzfeldt-Jakob disease therapies.
- Further development of diverse cellular models is critical for advancing prion disease therapeutics.
Related Concept Videos
03:03Amyloid Fibrils
11.5K
Amyloid fibrils are aggregates of misfolded proteins. Under most circumstances, misfolded proteins are either refolded by chaperone proteins or degraded by the proteasome. However, in the case of a mutation or a disease, these proteins can accumulate to form large clusters and often further assemble to form elongated fibers, called fibrils.
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining,...
Amyloid deposits were observed as early as 1639 in the liver and the spleen. In 1854, Rudolph Virchow performed iodine staining,...
11.5K
01:29Subviral Agents
407
Subviral agents are infectious entities that resemble viruses but lack one or more viral components, such as a capsid or essential replication machinery. These agents include viroids, prions, and satellites, each possessing distinct structural and functional characteristics that influence their mode of infection and replication.Viroids are the simplest subviral agents, consisting of circular, single-stranded RNA molecules without a protein coat. They exclusively infect plants, relying entirely...
407
01:21EPS and iPS Cells in Disease Research
3.3K
Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
3.3K